Top Cancer Stories of 2014

By Nancy Ciancaglini

2014 was an exciting year for cancer developments ­— from first-in-class FDA approvals for immune checkpoint inhibitors to obesity being identified as a leading cause of cancer. With no shortage of news, here is a recap of some of the headlines from the past year that generated a tsunami of media coverage.

1) First FDA Approvals for PD-1 Inhibitors—Expansion into Blood Cancers

Forbes named Keytruda and Opdivo “the most important drugs of 2014.” Keytruda (pembrolizumab, Merck) won an accelerated approval from the FDA on Sept. 4, 2014 for advanced melanoma in patients who no longer respond to other drugs, becoming the first anti-PD-1 immune checkpoint inhibitor to be approved by U.S. regulators. Opdivo (nivolumab, Bristol-Myers Squibb) followed with an FDA accelerated approval for the same indication on Dec. 22, 2014. These new immunotherapies act by releasing an immune system brake called PD-1 (programmed death receptor-1) that tumors use to fend off an immune system attack.

Researchers are excited because in 2014 both therapies demonstrated their clinical potential beyond solid tumors for treating blood cancers like Hodgkin lymphoma (HL). In early-stage data reported for the first time at ASH in December, patients with advanced HL who failed to respond to other currently approved therapies including Adcetris (brentuximab vedotin, Seattle Genetics) and stem cell transplants achieved partial or complete remissions of HL when treated with either Keytruda or Opdivo based on two separate small studies. The results were called ‘profound’ by investigators. Nivolumab has already been granted a “breakthrough therapy” designation in HL by the agency, which could speed its approval for the disease. Stay tuned for what’s hoped to be more good news on the clinical effectiveness of PD-1 therapies in treating a wide range of cancers, including difficult-to-treat blood cancers.

2) CAR-T Cell Therapies Are Hot

New data at ASH from a pediatric study in leukemia of Novartis’ investigational CAR therapy, CTL019, might signal a near-future sea change in the treatment of hematological malignancies. Researchers from the Children's Hospital of Philadelphia and the University of Pennsylvania provided longer-term and updated data from a small study of patients ages 5 to 22 with relapsed/refractory acute lymphocytic leukemia (ALL). Ninety-two percent, or 36 of 39 patients, had complete remissions of the disease after treatment with CTL019. Investigators reported that more than two-thirds of the patients were cancer free six months following treatment and 75% of patients were still alive. In a Lancet study published in October by a National Cancer Institute (NCI) team, researchers said that six out of 21 young patients with primary chemorefractory ALL who had never had a remission after multiple chemotherapy regimens showed a complete response after one infusion of CAR T-cell therapy. Clinicians think CAR-T cell therapy could replace stem cell transplant for some patients with blood cancers.

3) Power Morcellators and Hidden Cancer Risk

The backlash against the use of laparoscopic power morcellators was intense in 2014 and it divided doctors. After nearly a year of debate about the hidden cancer risk that the use of the tools posed to women during common gynecological procedures like uterine fibroid removal and hysterectomies in spreading undetected cancers like uterine sarcoma, the FDA took decisive action in November and issued an immediate “black box” warning for the devices. In April, the agency estimated that 1 in 350 women undergoing surgery for fibroids were at risk for an undetected cancer that the tool could spread and discouraged its use. But despite the FDA’s initial warning, reports surfaced in September that gynecologists continued to use the surgical tools. Johnson and Johnson (J&J), the U.S.’s biggest manufacturer of the devices, directed surgeons to stop using them and pulled them from the market, followed by a voluntary J&J worldwide recall in July. Hospitals, including Boston's Brigham and Women's Hospital and Philadelphia's Temple University Hospital, banned the use of the tools, and some insurers like Highmark stopped covering them. The FDA’s strongest warning in November came nearly a year after a review of the surgical cutting tools and is expected to sharply curtail their use.

4) The Continued Spiralling Costs of Cancer Drugs

“Financial toxicity” was a familiar phrase this past year, a side effect of the high cost of cancer care that leaves patients having to decide between either receiving life-saving treatments or personal bankruptcy. In a CBS “60 Minutes” interview in October, noted oncologist Leonard Saltz, MD, of Memorial Sloan-Kettering Cancer Center, pointed out that the cost of a new cancer drug is now well over $100K a year. Newly approved drugs proved the point: Blincyto (blinatumomab, Amgen) for ALL will cost around $178,000 a year, making it one of the world’s most expensive cancer drugs. Keytruda and Opdivo, both newly approved for advanced melanoma, will each carry a price tag of $150,000 per year. After the CBS piece aired, ASCO issued a statement saying that the organization was committed and working on several fronts to ensure that all patients would have access to high-quality, high-value cancer care.

5) New Payment Models for Cancer Care

Driven by the arrival of accountable care, alternative payment models to the traditional fee-for-service model in cancer care evolved in 2014. In August, the Center for Medicare and Medicaid Innovation (CMMI) released a preliminary design for an Oncology Care Model. ASCO has an initiative underway called the Consolidated Payments for Oncology Care, which will restructure how oncologists are paid. Wellpoint said it would pay oncologists a bonus of $350 per month per patient for following specified treatment regimens or “pathways,” saving 3% on overall cancer costs in the process. Florida Blue is teaming up with physician practices to create cancer-specific “accountable care organizations” that reward doctors if they save money and hit quality targets. And, in an expansion of a pilot project using flat payments for certain cancers, including drug costs, United Healthcare announced in December a pilot program with MD Anderson Cancer Center that would provide a “bundled payment” for head and neck cancers.